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UPDATE on ACE -031

"We are writing to let you know that Shire and Acceleron have decided to discontinue development of ACE-031 for Duchenne muscular dystrophy. As you may know, ACE-031 clinical trials have been on hold since February 2011. Since then, we have performed a number of additional non-clinical and toxicology studies, but unfortunately the findings from these studies do not support further development.

Thank you very much for your contributions to or interest in the ACE-031 program. We are deeply disappointed that we were not able to bring ACE-031 forward for the treatment of DMD patients. Each company remains committed to the development of novel therapies for DMD and other rare diseases."

Lawrence Charnas, MD, PhD Kenneth M. Attie, MD
Director & Head of Discovery Medicine VP, Medical Research
Shire Human Genetics Therapies Acceleron Pharm, Inc

ParentProjectMD's insight:

We received disappointing news today from Shire and Acceleron. In the first clinical trial of this drug in Duchenne, some participants experienced the serious adverse event of bleeding gums and telangiectasia (small dilated blood vessels near the surface of the skin). Since, Shire has spent considerable time in an effort to better understand the potential toxicity issues with the compound.


The good news is similar strategies that are less likely to cause toxicity continue to move toward clinical testing and Shire/Acceleron continue to be interested in Duchenne.

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Continued Access to Drisapersen for Patients Completing the US Ph IIb 48-week study

Continued Access to Drisapersen for Patients Completing the US Ph IIb 48-week study | Duchenne Research | Scoop.it

The purpose of this communication is provide an update to you, as you have requested, on GSK’s plans for providing the option of continued access to drisapersen for boys completing the US Ph IIb 48 week study (DMD114876) -clinicaltrials.gov.

ParentProjectMD's insight:

The latest update PPMD has received from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit.

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Pre-clinical efficacy data for WNT7A-analog program

Fate Therapeutics, Inc., a biopharmaceutical company engaged in the discovery and development of adult stem cell modulators, announced today the presentation of preclinical data from its WNT7a protein analog program for the treatment of muscular dystrophy at the MDA 2013 Scientific Conference, April 21-24, in Washington DC. The presentations describe the engineering of pharmaceutically optimized WNT7a protein analogs, as well as their mechanism of action and efficacy profile in preclinical pharmacology studies.

 

"We are working towards the nomination of an investigational new drug (IND) candidate later this year, and are excited about the potential to advance this new mechanism toward clinical studies." - Christian Weyer, President and CEO of Fate Therapeutics

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Catabasis Pharmaceuticals Announces Issuance of Patents for CAT-1000 and CAT-2000 Series

ParentProjectMD's insight:

These patents include a compound in development for Duchenne. A phase II study for Duchenne is being planned.

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Report from MDA's Scientific Conference 2013

Report from MDA's Scientific Conference 2013 | Duchenne Research | Scoop.it

While the content of the MDA meeting was wonderful,  it was equally important to engage in sidebar conversations with leaders from academia and industry.  Thomas Voit, MD is from France.  He has been in the field for many years and is clearly one of the most experienced and influential people in Europe.  We discussed a field that has changed and grown over the last 15 years, with projects seeded long ago with the hope that many would move into clinical trials and hopefully approvals.

ParentProjectMD's insight:

Most of what was presented MDA’s Scientific Conference are results that were previously announced, however there were some interesting presentations and results worth repeating that we wanted to share.

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Pre-clinical results on naproxcinod in Duchenne

Pre-clinical results on naproxcinod were presented at the MDA Scientific Conference in a poster entitled “Long-term treatment with naproxcinod significantly improves skeletal and cardiac disease phenotype in mdx mouse model of dystrophy." The effects of naproxcinod were investigated on skeletal and cardiac muscle function in mdx mice. The results of the study suggest that naproxcinod may have potential as a safe therapeutic option.

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